Correcting the NLRP3 inflammasome deficiency in macrophages from autoimmune NZB mice with exon skipping antisense oligonucleotides

Immunology and Cell Biology

Volumn 94, Issue 5, 2016, Pages 520-524

  Thygesen, Sara J ^a   Sester, David P ^a   Cridland, Simon O ^a   Wilton, Steve D ^b,c   Stacey, Katryn J ^a  

^a UNIVERSITY OF QUEENSLAND   (Australia)

^b Western Australian Research Inst   (Australia)

^c MURDOCH UNIVERSITY   (Australia)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; CRYOPYRIN; INFLAMMASOME; MESSENGER RNA; NLRP3 PROTEIN, MOUSE;

ANIMAL CELL; ARTICLE; AUTOIMMUNITY; BINDING SITE; BONE MARROW DERIVED MACROPHAGE; CELL VIABILITY; CODON; EXON SKIPPING; FEMALE; IN VIVO STUDY; INTRON; MOUSE; NEW ZEALAND BLACK MOUSE; NONHUMAN; POINT MUTATION; PROTEIN EXPRESSION; PYROPTOSIS; RNA SPLICING; SPLICING DEFECT; ALTERNATIVE RNA SPLICING; ANIMAL; C57BL MOUSE; DEFICIENCY; DRUG EFFECTS; EXON; GENETICS; MACROPHAGE; METABOLISM; NUCLEOTIDE SEQUENCE;

ALTERNATIVE SPLICING; ANIMALS; AUTOIMMUNITY; BASE SEQUENCE; EXONS; INFLAMMASOMES; MACROPHAGES; MICE, INBRED C57BL; MICE, INBRED NZB; NLR FAMILY, PYRIN DOMAIN-CONTAINING 3 PROTEIN; OLIGONUCLEOTIDES, ANTISENSE;

EID: 84956946745     PISSN: 08189641     EISSN: 14401711     Source Type: Journal    
DOI: 10.1038/icb.2016.3     Document Type: Article

References (32)

1. Martinon F, Burns K, Tschopp J. The inflammasome: a molecular platform triggering activation of inflammatory caspases and processing of proIL-beta. Mol Cell 2002; 10: 417-426
2. Ghayur T, Banerjee S, Hugunin M, Butler D, Herzog L, Carter A, et al. Caspase-1 processes IFN-gamma-inducing factor and regulates LPS-induced IFN-gamma production. Nature 1997; 386: 619-623
3. Thornberry NA, Bull HG, Calaycay JR, Chapman KT, Howard AD, Kostura MJ, et al. A novel heterodimeric cysteine protease is required for interleukin-1 beta processing in monocytes. Nature 1992; 356: 768-774
4. Sagulenko V, Thygesen SJ, Sester DP, Idris A, Cridland JA, Vajjhala PR, et al. AIM2 and NLRP3 inflammasomes activate both apoptotic and pyroptotic death pathways via ASC. Cell Death Differ 2013; 20: 1149-1160
5. Bauernfeind F, Hornung V. Of inflammasomes and pathogens-sensing of microbes by the inflammasome. EMBO Mol Med 2013; 5: 814-826
6. Mariathasan S, Weiss DS, Newton K, McBride J, O'Rourke K, Roose-Girma M, et al. Cryopyrin activates the inflammasome in response to toxins and ATP. Nature 2006; 440: 228-232
7. Dostert C, Petrilli V, Van Bruggen R, Steele C, Mossman BT, Tschopp J. Innate immune activation through Nalp3 inflammasome sensing of asbestos and silica. Science 2008; 320: 674-677
8. Sester DP, Sagulenko V, Thygesen SJ, Cridland JA, Loi YS, Cridland SO, et al. Deficient NLRP3 and AIM2 inflammasome function in autoimmune NZB mice. J Immunol 2015; 195: 1233-1241
9. Scatizzi JC, Haraldsson MK, Pollard KM, Theofilopoulos AN, Kono DH. The Lbw2 locus promotes autoimmune hemolytic anemia. J Immunol 2012; 188: 3307-3314
10. Borchers A, Ansari AA, Hsu T, Kono DH, Gershwin ME. The pathogenesis of autoimmunity in New Zealand mice. Semin Arthritis Rheum 2000; 29: 385-399
11. Aartsma-Rus A, van Ommen GJ. Antisense-mediated exon skipping: a versatile tool with therapeutic and research applications. RNA 2007; 13: 1609-1624
12. Long M, Deutsch M. Association of intron phases with conservation at splice site sequences and evolution of spliceosomal introns. Mol Biol Evol 1999; 16: 1528-1534
13. Coolidge CJ, Seely RJ, Patton JG. Functional analysis of the polypyrimidine tract in pre-mRNA splicing. Nucleic Acids Res 1997; 25: 888-896
14. Fairbrother WG, Yeh RF, Sharp PA, Burge CB. Predictive identification of exonic splicing enhancers in human genes. Science 2002; 297: 1007-1013
15. Yeo G, Hoon S, Venkatesh B, Burge CB. Variation in sequence and organization of splicing regulatory elements in vertebrate genes. Proc Natl Acad Sci USA 2004; 101: 15700-15705
16. Gallego-Villar L, Viecelli HM, Perez B, Harding CO, Ugarte M, Thony B, et al. A sensitive assay system to test antisense oligonucleotides for splice suppression therapy in the mouse liver. Mol Ther Nucleic Acids 2014; 3: e193
17. Karras JG, McKay RA, Dean NM, Monia BP. Deletion of individual exons and induction of soluble murine interleukin-5 receptor-Alpha chain expression through antisense oligonucleotide-mediated redirection of pre-mRNA splicing. Mol Pharmacol 2000; 58: 380-387
18. Aartsma-Rus A, Houlleberghs H, van Deutekom JC, van Ommen GJ, t Hoen PA. Exonic sequences provide better targets for antisense oligonucleotides than splice site sequences in the modulation of Duchenne muscular dystrophy splicing. Oligonucleotides 2010; 20: 69-77
19. Echigoya Y, Mouly V, Garcia L, Yokota T, Duddy W. In silico screening based on predictive algorithms as a design tool for exon skipping oligonucleotides in Duchenne muscular dystrophy. PLoS ONE 2015; 10: e0120058
20. Aartsma-Rus A, van Vliet L, Hirschi M, Janson AA, Heemskerk H, de Winter CL, et al. Guidelines for antisense oligonucleotide design and insight into splice-modulating mechanisms. Mol Ther 2009; 17: 548-553
21. Harding PL, Fall AM, Honeyman K, Fletcher S, Wilton SD. The influence of antisense oligonucleotide length on dystrophin exon skipping. Mol Ther 2007; 15: 157-166
22. Fletcher S, Adams AM, Johnsen RD, Greer K, Moulton HM, Wilton SD. Dystrophin isoform induction in vivo by antisense-mediated alternative splicing. Mol Ther 2010; 18: 1218-1223
23. Sazani P, Kole R. Therapeutic potential of antisense oligonucleotides as modulators of alternative splicing. J Clin Invest 2003; 112: 481-486
24. Krawczak M, Reiss J, Cooper DN. The mutational spectrum of single base-pair substitutions in mRNA splice junctions of human genes: causes and consequences. Hum Genet 1992; 90: 41-54
25. Sterne-Weiler T, Howard J, Mort M, Cooper DN, Sanford JR. Loss of exon identity is a common mechanism of human inherited disease. Genome Res 2011; 21: 1563-1571
26. Veltrop M, Aartsma-Rus A. Antisense-mediated exon skipping: taking advantage of a trick from Mother Nature to treat rare genetic diseases. Exp Cell Res 2014; 325: 50-55
27. Koo T, Wood MJ. Clinical trials using antisense oligonucleotides in duchenne muscular dystrophy. Hum Gene Ther 2013; 24: 479-488
28. Cirak S, Arechavala-Gomeza V, Guglieri M, Feng L, Torelli S, Anthony K, et al. Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study. Lancet 2011; 378: 595-605
29. Sester DP, Brion K, Trieu A, Goodridge HS, Roberts TL, Dunn J, et al. CpG DNA activates survival in murine macrophages through TLR9 and the phosphatidylinositol 3-kinase-Akt pathway. J Immunol 2006; 177: 4473-4480
30. Austyn JM, Gordon S. F4/80, a monoclonal antibody directed specifically against the mouse macrophage. Eur J Immunol 1981; 11: 805-815
31. Cridland JA, Curley EZ, Wykes MN, Schroder K, Sweet MJ, Roberts TL, et al. The mammalian PYHIN gene family: phylogeny, evolution and expression. BMC Evol Biol 2012; 12: 140
32. Berridge MV, Tan AS. Characterization of the cellular reduction of 3-(4, 5-dimethylthiazol-2-yl)-2, 5-diphenyltetrazolium bromide (MTT): subcellular localization, substrate dependence, and involvement of mitochondrial electron transport in MTT reduction. Arch Biochem Biophys 1993; 303: 474-482